Sionna Therapeutics

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Sionna Therapeutics is on a mission to revolutionize cystic fibrosis treatment by developing novel small molecules that directly stabilize the NBD1 domain of the CFTR protein, the site of the most common disease-causing mutation. Leveraging over a decade of foundational research, the company has advanced a pipeline of NBD1 stabilizers and complementary modulators into clinical development, aiming to normalize CFTR function where current standards of care fall short. Its strategy is to build a dominant CF franchise by developing these candidates as potential best-in-class therapies, either alone or in combination, to address the significant unmet need for patients who remain underserved by existing modulator regimens.

A proprietary platform for the rational design of small molecule NBD1 stabilizers, leveraging structural biology, biophysical screening, and predictive CF human bronchial epithelial cell models to correct the root cause of CFTR protein dysfunction in F508del cystic fibrosis.